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BCX4161

Phase 2

Hereditary Angioedema | Small molecule | Immunology |BioCryst Pharmaceuticals, Inc.|Last Updated: Nov 17, 2025

Success Probability

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials4
Total Enrollment169

FDA Designations

No designations recorded

Clinical trial landscape

BCX4161 · 4 trials · 2 indications

Phase 2 2Phase 1 2
NCT0230362612-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE AttacksHereditary Angioedema
COMPLETED110 Analytics
NCT01984788Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAEHereditary Angioedema
COMPLETED24 Analytics
PHASE2COMPLETED
12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks
Hereditary AngioedemaUnlock trial analytics
PHASE2COMPLETED
Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE
Hereditary AngioedemaUnlock trial analytics

Study Endpoints

Primary Endpoints

The Mean Acute Angioedema Attack Rate
12 weeks

An angioedema attack was defined as swelling at any location reported by participant, which had no swelling earlier. Total number of confirmed attacks during the treatment period standardized to a weekly attack-rate to adjust for the total duration of treatment. The attack rate was derived for each participant by treatment period. The weekly attack rate was equal to the total number of confirmed attacks during a treatment period divided by the duration of the treatment (in days) times 7 days.

Number of acute angioedema attacks
Over 28 days of treatment
Mass balance of BCX4161 after a single oral dose of [14C] BCX4161 based upon radioactivity excreted in urine, expired air and feces
Determined from samples drawn up to 14 days post-dose
Plasma pharmacokinetics to assess the relative bioavailability of BCX4161 soft gelatin capsules to hard gelatin capsules
Pharmacokinetic parameters AUC0-inf, AUC0-t and Cmax generated after 24 hours of postdose blood sampling

Secondary Endpoints

Number of Attack-free Days
12 weeks
Number of Participants Who Are Attack-free
12 weeks
Disease Activity, as Measured by the 84-day Angioedema Activity Score
12 weeks
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposePREVENTION

Treatment Arms

ArmTypeDescription
BCX4161 300 mg three times dailyEXPERIMENTALThree BCX4161 capsules (100 mg) and two placebo capsules to be taken three times daily by mouth
BCX4161 500 mg three times dailyEXPERIMENTALFive BCX4161 capsules (100 mg) to be taken three times daily by mouth
Placebo three times dailyPLACEBO_COMPARATORFive placebo capsules to be taken three times daily by mouth
BCX4161ACTIVE_COMPARATOR400 mg TID for 28 days
PlaceboPLACEBO_COMPARATORTID for 28 days
[14C] BCX4161EXPERIMENTALIncludes a radiolabelled dose of \[14C\] BCX4161 and unlabelled BCX4161
Treatment AEXPERIMENTALBCX4161 400 mg formulated as hard gelatin capsules given orally under fasting conditions x1
Treatment BEXPERIMENTALBCX4161 400 mg formulated as soft gelatin capsules given orally under fasting conditions x1
Treatment CEXPERIMENTALBCX4161 400 mg formulated as soft gelatin capsules given orally after a high-fat breakfast x1

Interventions

NameTypeDescription
BCX4161DRUG -
PlaceboDRUG -
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites40

Key Inclusion Criteria: 1. A clinical diagnosis of HAE type I or II 2. Documented HAE attacks within a defined calendar period; in the absence of documented HAE attacks, participants will be required to enter a run-in period to document attacks 3. Access to acute attack medications 4. Sexually acti...

Countries:United StatesBelgiumCanadaFranceGermanyHungaryItalyUnited Kingdom
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Frequently asked questions about BCX4161

What is BCX4161 used for?

BCX4161 is an investigational small molecule being developed for hereditary angioedema (HAE), a condition characterized by recurrent episodes of severe swelling. It is being studied as an oral prophylactic treatment to prevent HAE attacks. The drug is in Phase 2 clinical development and is not yet approved by regulatory authorities.

Who makes BCX4161?

BCX4161 is being developed by BioCryst Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol BCRX. The company is conducting clinical trials to evaluate the safety and efficacy of BCX4161 as a prophylactic treatment for hereditary angioedema.

What phase is BCX4161 in?

BCX4161 is in Phase 2 clinical development for hereditary angioedema. It has completed four clinical trials, including two Phase 2 studies and two Phase 1 studies. The drug is investigational and has not received FDA approval, meaning it is still being evaluated in clinical trials.

What clinical trials is BCX4161 in?

BCX4161 has completed four clinical trials. These include NCT01984788, a Phase 2 safety and efficacy study as prophylaxis for HAE; NCT02125162, a Phase 1 bioavailability study; NCT02218294, a Phase 1 metabolism study; and NCT02303626, a 12-week Phase 2 study of oral prophylaxis against HAE attacks.

Is BCX4161 FDA approved?

BCX4161 is not FDA approved. It is an investigational drug currently in Phase 2 clinical development for hereditary angioedema. All four of its clinical trials have been completed, but the drug has not yet received regulatory approval and remains under investigation for safety and efficacy.